Clinical Trials Logo

Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT03933410
Other study ID # K020-218
Secondary ID
Status Terminated
Phase Phase 2
First received
Last updated
Start date September 17, 2019
Est. completion date March 2, 2021

Study information

Verified date January 2022
Source Kaleido Biosciences
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

UNLOCKED: A Phase 2 Trial to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care


Description:

We expect the trial to enroll approximately 24 Urea Cycle Disorder (UCD) patients on standard of care with elevated ammonia levels. The planned treatment duration is eight weeks, with a primary endpoint of proportion of subjects who achieve a ≥15% reduction from baseline in fasting ammonia at the end of treatment. Patients will also be followed for safety and tolerability. This clinical trial is intended to allow us to evaluate efficacy of KB195 in reducing ammonia in UCD patients.


Recruitment information / eligibility

Status Terminated
Enrollment 13
Est. completion date March 2, 2021
Est. primary completion date February 2, 2021
Accepts healthy volunteers No
Gender All
Age group 18 Years to 65 Years
Eligibility Key Inclusion Criteria: - Signed informed consent and willing to comply with protocol-specified procedures. - Has any confirmed UCD other than N-acetyl glutamatesynthase (NAGS) deficiency. - Is male or female, 12 to 70 years of age (inclusive) - If = 18 years old, has a BMI =20.0 and < 40.0 kg/m2. If < 18 years old, has a BMI between 5th percentile and 95th percentile and weight greater than 5th percentile according to age, sex and regionally appropriate growth chart - Has evidence of poorly controlled disease on the current standard of care (SOC) - If NBT is part of SOC, is on a stable dose and regimen for at least 4 weeks before Screening and the dose is expected to remain stable during the study - Is willing to maintain a stable diet throughout the course of study and is willing to continue usual exercise routine. - If taking probiotics or prebiotics, is on a stable dose regimen for at least 4 weeks before Screening and the dose and regimen are expected to remain stable during the study - Has a negative urine screen for drugs of abuse at Screening - If male or female of child bearing potential, agree with use effective method of contraception for the duration of the study and 90 days after last dose of study product Key Exclusion Criteria: - Is at a high risk for metabolic decomposition. - Has had a substantive change in diet or any other aspect of UCD management within 4 weeks before the Screening Visit - Has used a systemic anti-infective within 4 weeks before the Screening Visit, or use is anticipated during the study - Has been diagnosed with Citrullinemia Type II - Is receiving any systemically administered immunosuppressant medication on a chronic basis - Has changed the use of or dose of any drug or other compound to modulate GI motility within 4 weeks before the Screening Visit, or the use or dose is expected change during the course of the study - Has a history of or active GI or liver disease - Has a prior solid organ transplantation including liver transplantation, or is anticipated to receive a liver transplant during study participation - Has used an investigational drug, product, or device within 30 days before the Screening Visit - Has a contraindication, sensitivity, or known allergy to the study drug - Is considered, in the opinion of the PI, to likely be a poor attendee or unlikely for any reason to be able to comply with the study drug procedures

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
KB195
KB195 is a novel glycan

Locations

Country Name City State
Belgium Cliniques Universitaires Saint-Luc Bruxelles
Belgium Universitair Ziekenhuis Gent Gent
Germany Zentrum für Kinder- und Jugendmedizin Angelika-Lautenschläger-Klinik Heidelberg
Spain Hospital de Cruces Barakaldo
Spain Hosptial Universitari Vall d'Hebron Barcelona
Spain Hospital Universitario Reina Sofia Córdoba
Spain Hospital Universitario Virgen del Rocio Sevilla
Switzerland Inselpital, Universitaetsklinik fur Kinderheikunde Bern
Switzerland Inselpital, Universitätsklinik für Kinderheilkunde Bern
Turkey Department of Child Health and Diseases, Department of Nutrition and Metabolism Istanbul University Istanbul
United Kingdom National Hospital for Neurology and Neurosurgery London
United Kingdom Salford Royal Hospital Salford Greater Manchester
United Kingdom Salford Royal NHS Foundation Trust Salford
United States Johns Hopkins University School of Medicine Baltimore Maryland
United States Icahn School of Medicine at Mount Sinai-Clinical Research Unit New York New York
United States UPMC Children's Hospital of Pittsburgh Pittsburgh Pennsylvania
United States Washington University School of Medicine Saint Louis Missouri
United States University of South Florida/ USF HEALTH Tampa Florida
United States Children's National Medical Center Washington District of Columbia

Sponsors (1)

Lead Sponsor Collaborator
Kaleido Biosciences

Countries where clinical trial is conducted

United States,  Belgium,  Germany,  Spain,  Switzerland,  Turkey,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Proportion of subjects who achieve a =15% reduction from baseline in fasting plasma ammonia at the end of treatment. Day -1 to Day 55
Secondary Proportion of subjects normalizing their fasting plasma ammonia concentrations from above the upper limit of normal at baseline to below the upper limit of normal at the end of treatment. Day -1 to Day 55
Secondary Number of subjects experiencing adverse events (AEs) Day -28 to Day 84
Secondary Number of subjects experiencing severe adverse events (SAEs) Day -28 to Day 84
Secondary Change from baseline to end of treatment in Gastrointestinal Tolerability Questionnaire (GITQ) scores Evaluate the effect of KB195 on self-report questionnaires including the Gastrointestinal Tolerability Questionnaire, an assessment of the frequency and severity of GI symptoms, e.g., gas, abdominal pain, calculated on a scale from 0 (None/Not applicable) to a maximum score of 60 (Severe/Much more than usual) for all questions Day -28 to Day 84
Secondary Change from baseline to end of treatment in Bristol Stool Scale (BSS) scoring. Evaluate the effect of KB195 on self-report questionnaires including the Bristol Stool Scale, an assessment of stool consistency on a scale from 1 (separate hard lumps, like nuts, hard to pass) through 7 (watery, no solid pieces, entirely liquid) Day -28 to Day 84
See also
  Status Clinical Trial Phase
Terminated NCT01948427 - Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
Completed NCT03064048 - Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD N/A
Recruiting NCT04612764 - Liver Disease in Urea Cycle Disorders
Completed NCT05330039 - Characterization of Intestinal Microbiota in Children With Inborn Errors of Metabolism (IEM)
Completed NCT03721367 - Chronic Liver Disease in Urea Cycle Disorders
Completed NCT03911089 - A Collection of Case Studies in Infants With UCD to Evaluate Infant Growth and the Safety of a New Medical Food for UCD N/A
Terminated NCT03181828 - Manipulating the Gut Microbiome Study Phase 1/Phase 2
Completed NCT00472732 - Neurologic Injuries in Adults With Urea Cycle Disorders N/A
Recruiting NCT05076318 - Dysregulated Urea-synthesis at Terminal Uremia N/A
Completed NCT03179878 - Safety and Tolerability of SYNB1020-CP-001 Phase 1
Recruiting NCT04602325 - Systemic Biomarkers of Brain Injury From Hyperammonemia
Withdrawn NCT03884959 - A Safety and Efficacy Study of Infusions of HepaStem in Urea Cycle Disorders Pediatric Patients Phase 2
Recruiting NCT04908319 - Hepatic Histopathology in Urea Cycle Disorders
Completed NCT03335488 - Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs) Phase 4
Completed NCT02246218 - A Study of the Safety, Efficacy and Pharmacokinetics of Glycerol Phenylbutyrate in Pediatric Subjects Under 2 Years of Age With Urea Cycle Disorders Phase 4
Completed NCT04248062 - Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism
Completed NCT05706714 - Th1, Th2, Th17 Phenotype in Urea Cycle Disorders
Terminated NCT03343756 - HepaStem Long-Term Safety Registry